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NCT05432310 · ClinicalTrials.gov registry record · Phase 1
Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector
A Phase 1 study of Adenosine Deaminase Severe Combined Immune Deficiency, sponsored by University of California, Los Angeles.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 20
- Enrollment target
- 1
- Study location
NCT05432310 is a Phase 1 study of Adenosine Deaminase Severe Combined Immune Deficiency that is actively recruiting participants, run by University of California, Los Angeles. The registered enrollment target is 20 participants. The trial reports 1 study location across 1 state.
The verdict
NCT05432310, a Phase 1 study of Adenosine Deaminase Severe Combined Immune Deficiency, is actively recruiting participants, sponsored by University of California, Los Angeles.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 20 participants
- Enrollment target
- 1
- Study location
Study Summary
The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human ADA gene transfer by the EFS-ADA lentiviral vector. The level of gene transfer in blood cells and immune function will be measured as endpoints.
Conditions Studied
Interventions
- COMBINATION_PRODUCT A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme
Study Locations (1)
California
- University of California, Los Angeles (UCLA) - Los Angeles
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2023-01-04 |
| Est. Completion | 2026-12-31 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05432310
The ClinicalTrials.gov registry entry for NCT05432310 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 20 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is University of California, Los Angeles, which has 897 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Adenosine Deaminase Severe Combined Immune Deficiency appearing as the primary indexed condition, and to 1 intervention - of which A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme is the first listed.
NCT05432310 reports 1 study location spanning 1 distinct geographic area - top geographies include California.
Frequently Asked Questions
What is clinical trial NCT05432310 about?
NCT05432310 is a clinical study titled "Gene Therapy for Adenosine Deaminase Severe Combined Immune Deficiency Using Peripheral Blood and EFS ADA Vector". The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral blood (mPB) of ADA-deficient SCID infants and children following human ADA gene transfer by the EFS-ADA lentiviral vector. The level of gene tr...
What is the current status of trial NCT05432310?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 20 participants. The study started on 2023-01-04. Estimated completion is 2026-12-31.
What conditions does trial NCT05432310 study?
This clinical trial studies the following conditions: Adenosine Deaminase Severe Combined Immune Deficiency.
What interventions are being tested in trial NCT05432310?
The interventions under investigation include: A cryopreserved formulation of autologous mPB CD34+ hematopoietic stem and progenitor cells transduced ex vivo with the EFS-ADA lentiviral vector encoding the human ADA enzyme (COMBINATION_PRODUCT).
Who is sponsoring clinical trial NCT05432310?
This trial is sponsored by University of California, Los Angeles, which has 897 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05432310 being conducted?
This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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